Clinical trial guidelines for pharmacological treatment of irritable bowel syndrome

作者：Enrico Stefano Corazziari, Peter M. Bytzer, Michael Delvaux, Gerald Holtmann, J.‐R. Malagelada, James A. Morris, Stefan A Müller-Lissner, Robert E. Spiller, Jan F. Tack, Peter James Whorwell · 发表于：Alimentary Pharmacology & Therapeutics · 年份：2003 · DOI：10.1046/j.1365-2036.2003.01709.x · 被引用次数：86 · 研究领域：Gastrointestinal motility and disorders、Complementary and Alternative Medicine Studies、Pathogenesis and Treatment of Hiccups

Appropriate guidelines for clinical trials in irritable bowel syndrome are needed because of the inadequacy of previously performed trials, the use of new and more adequate patient definition, new emerging pathophysiological models and the unique requirements related to the assessment of treatment outcome that, in the absence of a biological marker, can rely only on the evaluation of clinical manifestations. This consensus report highlights the following points. (a) A 4-week period is considered to be adequate to assess drug efficacy for the control of symptoms. (b) For the cyclic and non-life-threatening nature of the disease, a long-term study of 4-6 months or more of active treatment to establish efficacy is considered to be inappropriate in the large majority of patients. (c) In the initial assessment phase of drug efficacy, the withdrawal effect of treatment can be ascertained during a follow-up period prolonged for a sufficient time (4-8 weeks) after stopping treatment. Subsequent trials with proper withdrawal phase design and duration can then ascertain the drug post-treatment benefit. (d) Considering the intermittent clinical manifestations of irritable bowel syndrome, designing trials with on-demand or repeated cycles of treatment could be envisaged. However, the lack of a definition of what constitutes an exacerbation is a major obstacle to the design of such trials. In the absence of an established gold standard, appropriately justified novel trial designs are welc...